for males ages 10 years and up (full criteria)
at UC Davis
study started
estimated completion
Principal Investigator
by Craig McDonald, MD (ucdavis)



HOPE-3 is a multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period. All participants will be eligible to receive CAP-1002 for an additional 12 months as part of an open label extended safety assessment period.

Official Title

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy


Up to 68 eligible study participants will be randomized to either CAP-1002 or placebo in a 1:1 ratio. The trial will include visits at Screening, Baseline/Day 1, Week 4, and Months 3, 6, 9, and 12 with IV infusions of CAP-1002 or placebo on Day 1 and Months 3, 6, and 9. The primary analysis of efficacy and safety will be performed at Month 12 following 4 administrations of CAP-1002 or placebo. The primary efficacy endpoint is the mean change from baseline in upper limb function as assessed by the full Performance of the Upper Limb test, version 2.0 [PUL 2.0] at the 12-month time point. Secondary endpoints evaluated at the 12-month time point include assessment of changes in cardiac muscle function and structure by cardiac magnetic resonance imaging [cMRI], changes in hand-to-mouth function [eat 10-bites assessed by the Duchenne Video Assessment (DVA)], and quality of life assessments. Safety evaluations will include adverse events, concomitant medications, physical exam, vital signs, 12-lead ECG, and clinical laboratory testing. Following the initial 4 infusions, all subjects will be eligible to participate in a 12-month open label extended safety assessment period and receive up to 4 additional IV infusions of CAP-1002 once every 3 months. An analysis of extended safety will be conducted after all subjects have completed the Month 24 visit.


Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Neuromuscular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Nervous System Diseases Duchenne Muscular Dystrophy Cell Therapy Performance of the Upper Limb Ambulatory Non-Ambulatory CAP-1002


You can join if…

Open to males ages 10 years and up

  1. Male subjects at least 10 years of age at time of consent who are willing and able to provide informed consent to participate in the trial and diagnosed with DMD as confirmed by the Investigator
  2. Genetically confirmed DMD
  3. Performance of the Upper Limb test (PUL) entry item scores 2-6 and total PUL score less than or equal to 40
  4. Reduced ability to walk/run (if ambulatory): 10-meter walk/run velocity < 1 meter/second and total PUL score of less than or equal to 40
  5. Loss of independent ambulation between 10th and 18th year birthday
  6. Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for weight-based or toxicity-related adjustments
  7. Current and up-to-date immunizations
  8. Adequate venous access for parenteral IP infusions and routine blood collection
  9. Assessed by the Investigator as willing and able to comply with the requirements of the trial

You CAN'T join if...

  1. Left ventricular ejection fraction (LVEF) < 35%
  2. Elbow-flexion contractures > 30° in both extremities
  3. Body mass index (BMI) > 45
  4. Percent predicted forced vital capacity (FVC%) < 35%
  5. Inability to perform consistent PUL 2.0 measurement within ± 2 points without shoulder domain or within ± 3 points with shoulder domain during paired testing at screening
  6. Risk of near-term respiratory decompensation in the judgment of the Investigator, or the need for initiation of day and night non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L at screening
  7. History of non DMD-related chronic respiratory disease requiring ongoing or intermittent treatment, including, but not limited to, asthma, bronchitis, and tuberculosis
  8. Acute respiratory illness within 30 days prior to screening and during screening
  9. Initiation of nocturnal non-invasive ventilation within 30 days prior to screening
  10. . Planned or anticipated thoracic or spinal surgery within the 6 months following randomization
  11. . Planned or anticipated lower extremity surgery within the 6 months following randomization, if ambulatory
  12. . Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products
  13. . Initiation of treatment with metformin or insulin within 3 months prior to randomization
  14. . Initiation of treatment with an FDA-approved exon skipping therapy for the treatment of DMD within 24 months prior to randomization or dose adjustments to the therapy within 12 months prior to randomization with the exception of weight-based dose adjustments
  15. . Treatment with human growth hormone within 3 months prior to randomization, unless on a stable dose allowing for weight-based dose adjustments (as determined by the site Investigator) for at least 24 months prior to randomization
  16. . Treatment with a cell therapy product within 12 months prior to randomization
  17. . Treatment with an investigational product within 6 months prior to randomization
  18. . History, or current use, of drugs or alcohol that could impair the ability to comply with participation in the trial
  19. . Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator


  • University of California, Davis not yet accepting patients
    Sacramento California 95817 United States
  • Arkansas Children's Hospital accepting new patients
    Little Rock Arkansas 72202 United States

Lead Scientist at University of California Health

  • Craig McDonald, MD (ucdavis)
    Professor, Physical Medicine and Rehabilitation. Authored (or co-authored) 214 research publications


accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
Capricor Inc.
Phase 3 research study
Study Type
Expecting 68 study participants
Last Updated